Inventiva, a biopharmaceutical company developing innovative therapies, particularly in fibrosis, announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to odiparcil (formerly IVA336) for the treatment of MPS VI.
“This decision could accelerate the availability of a much needed new treatment for MPS VI patients,” said Christine Lavery, President of the UK MPS Society.
“Odiparcil has the potential to become a breakthrough treatment for patients affected with MPS VI and I am looking forward to the upcoming phase IIa clinical trial which could prove the efficacy of odiparcil as a stand-alone treatment,” added Professor Chris Hendriksz, of FYMCA Medical Ltd. and University of Pretoria, South Africa.
“We recently received U.S. orphan drug status and with this new EU designation we continue delivering on our regulatory strategy for odiparcil. Clearly the recent preclinical data we generated showing that odiparcil is active in organs where marketed enzyme replacement therapies have limited or no efficacy, has been instrumental in convincing regulators to grant these ODD designations. These designations confirm that regulatory agencies share our view that odiparcil could improve MPS VI current treatment options,” concluded Pierre Broqua, Chief Scientific Officer and Co-Founder of Inventiva.
MPS VI (Maroteaux-Lamy syndrome), is a rare pediatric genetic degenerative disease with a prevalence estimated to be 1 in 225,000 live births. There is no cure for MPS VI and current treatment options, such as enzyme replacement therapy (ERT) or hematopoietic stem cell transplant (HSCT), leave the patients with high unmet medical needs.
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